Advancing Mitochondrial Health in Huntington Disease (HD): Small Molecule Therapies and Neurodegeneration.

Q3 Medicine
Vasanth S, Rakhi Mishra, Subhashree Sahoo, Sadia Parveen, Zuber Khan, Mumtaz, Ruqaiya, Rahul Pal
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Abstract

Huntington's disease (HD) is a severe neurodegenerative disorder caused by an expanded CAG repeat in the huntingtin gene, leading to the production of a mutant huntingtin protein. This mutation results in progressive motor, cognitive, and psychiatric impairments, alongside significant neuronal loss. Mitochondrial dysfunction plays a pivotal role in the pathophysiology of HD, contributing to disease progression and neuronal death. This article aims to evaluate small molecule-based therapeutic strategies designed to enhance mitochondrial function as a potential approach to alleviate symptoms and slow the progression of HD and related neurodegenerative disorders. A comprehensive review of recent literature is conducted to identify small molecules targeting mitochondrial dysfunction from Google Scholar, Pub- Med/Medline/PMC, ScienceDirect, Elsevier, Google Patents, and Clinicaltrials.gov.in, among others. The analysis focuses on their mechanisms of action, including reducing oxidative stress, enhancing mitochondrial biogenesis, and improving mitochondrial dynamics and function. The review identifies several promising small molecules capable of targeting mitochondrial dysfunction. These agents demonstrate potential in preclinical studies to alleviate HD symptoms and modify disease progression by addressing key aspects of mitochondrial health. Small molecule therapies targeting mitochondrial dysfunction offer considerable promise for treating HD. However, further research is required to optimize these therapies for clinical use and to evaluate their long-term impact on disease progression to fully establish their therapeutic efficacy.

推进亨廷顿病(HD)的线粒体健康:小分子治疗和神经变性。
亨廷顿氏病(HD)是一种严重的神经退行性疾病,由亨廷顿蛋白基因中CAG重复扩增引起,导致亨廷顿蛋白突变。这种突变导致进行性运动、认知和精神障碍,并伴有显著的神经元损失。线粒体功能障碍在HD的病理生理中起着关键作用,有助于疾病进展和神经元死亡。本文旨在评估旨在增强线粒体功能的基于小分子的治疗策略,作为缓解HD和相关神经退行性疾病症状和减缓进展的潜在途径。我们对谷歌Scholar、Pub- Med/Medline/PMC、ScienceDirect、Elsevier、谷歌Patents和Clinicaltrials.gov.in等机构的近期文献进行了全面的综述,以确定针对线粒体功能障碍的小分子。重点分析其作用机制,包括减少氧化应激,促进线粒体生物发生,改善线粒体动力学和功能。该综述确定了几种有希望的靶向线粒体功能障碍的小分子。这些药物在临床前研究中显示出通过解决线粒体健康的关键方面来缓解HD症状和改变疾病进展的潜力。针对线粒体功能障碍的小分子疗法为治疗HD提供了相当大的希望。然而,需要进一步的研究来优化这些治疗方法的临床应用,并评估其对疾病进展的长期影响,以充分确定其治疗效果。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
Current aging science
Current aging science Medicine-Geriatrics and Gerontology
CiteScore
3.90
自引率
0.00%
发文量
40
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